Patients' preferences for treatment outcomes for advanced non-small cell lung cancer: a conjoint analysis.
ABSTRACT Treatment decisions for advanced non-small cell lung cancer (NSCLC) are complex and require trade-offs between the benefits and risks experienced by patients. We evaluated the benefits that patients judged sufficient to compensate for the risks associated with therapy for NSCLC.
Participants with a self-reported diagnosis of NSCLC (n=100) were sampled from an online panel in the United Kingdom. Eligible and consenting participants then completed a self-administered online survey about their disease and their treatment preferences were assessed. This involved respondents choosing among systematically paired profiles that spanned eight attributes: progression-free survival [PFS], symptom severity, rash, diarrhoea, fatigue, nausea and vomiting, fever and infection, and mode of treatment administration (infusion and oral). A choice model was estimated using mixed-logit regression. Estimates of importance for each attribute level and attribute were then calculated and acceptable tradeoffs among attributes were explored.
A total of 89 respondents (73% male) completed all choice tasks appropriately. Increases in PFS together with improvements in symptom severity were judged most important and increased with PFS benefit - 4 months: 5.7; 95% CI: 3.5-7.9; 5 months: 7.1; 95% CI: 4.4-9.9; and 7 months: 10.0; 95% CI: 6.1-13.9. However, improvements in PFS were viewed as most beneficial when disease symptoms were mild and as detrimental when patients had severe symptoms. Fatigue (5.0; 95% CI: 2.7-7.3) was judged to be the most important risk, followed by diarrhoea (2.8; 95% CI: 0.7-4.9), nausea and vomiting (2.1; 95% CI: 0.1-4.1), fever and infection (2.1; 95% CI: 0.2-4.1), and rash (2.0; 95% CI: 0.2-3.9). Oral administration was preferred to infusion (1.8; 95% CI: 0.0-3.6). Patients with mild and moderate symptoms traded PFS for less risks or more convenience if the severe symptoms were not experienced.
This study demonstrates the value of conjoint analysis in the study of patient preferences for cancer treatments. In this small sample of patients with NSCLC from the UK, we demonstrate that the value of improvements in PFS is conditional upon the severity of disease symptoms; and that risks are valued differently.
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ABSTRACT: Discrete choice experiments (DCEs) are used to elicit preferences of current and future patients and healthcare professionals about how they value different aspects of healthcare. Risk is an integral part of most healthcare decisions. Despite the use of risk attributes in DCEs consistently being highlighted as an area for further research, current methods of incorporating risk attributes in DCEs have not been reviewed explicitly. This study aimed to systematically identify published healthcare DCEs that incorporated a risk attribute, summarise and appraise methods used to present and analyse risk attributes, and recommend best practice regarding including, analysing and transparently reporting the methodology supporting risk attributes in future DCEs. The Web of Science, MEDLINE, EMBASE, PsycINFO and Econlit databases were searched on 18 April 2013 for DCEs that included a risk attribute published since 1995, and on 23 April 2013 to identify studies assessing risk communication in the general (non-DCE) health literature. Healthcare-related DCEs with a risk attribute mentioned or suggested in the title/abstract were obtained and retained in the final review if a risk attribute meeting our definition was included. Extracted data were tabulated and critically appraised to summarise the quality of reporting, and the format, presentation and interpretation of the risk attribute were summarised. This review identified 117 healthcare DCEs that incorporated at least one risk attribute. Whilst there was some evidence of good practice incorporated into the presentation of risk attributes, little evidence was found that developing methods and recommendations from other disciplines about effective methods and validation of risk communication were systematically applied to DCEs. In general, the reviewed DCE studies did not thoroughly report the methodology supporting the explanation of risk in training materials, the impact of framing risk, or exploring the validity of risk communication. The primary limitation of this review was that the methods underlying presentation, format and analysis of risk attributes could only be appraised to the extent that they were reported. Improvements in reporting and transparency of risk presentation from conception to the analysis of DCEs are needed. To define best practice, further research is needed to test how the process of communicating risk affects the way in which people value risk attributes in DCEs.The patient 02/2014; · 1.57 Impact Factor
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ABSTRACT: Background There is growing agreement that regulators performing benefit–risk evaluations should take patients’ and caregivers’ preferences into consideration. The Patient-Focused Drug Development Initiative at the US Food and Drug Administration offers patients and caregivers an enhanced opportunity to contribute to regulatory processes by offering direct testimonials. This process may be advanced by providing scientific evidence regarding treatment preferences through engagement of a broad community of patients and caregivers. Objective In this article, we demonstrate a community-engaged approach to measure caregiver preferences for potential benefits and risks of emerging therapies for Duchenne muscular dystrophy (DMD). Methods An advocacy oversight team led the community-engaged study. Caregivers’ treatment preferences were measured by using best–worst scaling (BWS). Six relevant and understandable attributes describing potential benefits and risks of emerging DMD therapies were identified through engagement with advocates (n = 5), clinicians (n = 9), drug developers from pharmaceutical companies and academic centers (n = 11), and other stakeholders (n = 5). The attributes, each defined across 3 levels, included muscle function, life span, knowledge about the drug, nausea, risk of bleeds, and risk of arrhythmia. Cognitive interviewing with caregivers (n = 7) was used to refine terminology and assess acceptability of the BWS instrument. The study was implemented through an online survey of DMD caregivers, who were recruited in the United States through an advocacy group and snowball sampling. Caregivers were presented with 18 treatment profiles, identified via a main-effect orthogonal experimental design, in which the dependent variable was the respondents’ judgment as to the best and worst feature in each profile. Preference weights were estimated by calculating the relative number of times a feature was chosen as best and as worst, which were then used to estimate relative attribute importance. Results A total of 119 DMD caregivers completed the BWS instrument; they were predominately biological mothers (67.2%), married (89.9%), and white (91.6%). Treatment effect on muscle function was the most important among experimental attributes (28.7%), followed by risk of heart arrhythmia (22.4%) and risk of bleeding (21.2%). Having additional postapproval data was relatively the least important attribute (2.3%). Conclusions We present a model process for advocacy organizations aiming to promote patient-centered drug development. The community-engaged approach was successfully used to develop and implement a survey to measure caregiver preferences. Caregivers were willing to accept a serious risk when balanced with a noncurative treatment, even absent improvement in life span. These preferences should inform the Food and Drug Administration’s benefit–risk assessment of emerging DMD therapies. This study highlights the synergistic integration of traditional advocacy methods and scientific approach to quantify benefit–risk preferences.Clinical Therapeutics. 01/2014; 36(5):624–637.
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ABSTRACT: Disease symptom management in patients with advanced non-small cell lung cancer (NSCLC) is a critical aspect of therapy. The main objective of our study was to assess patient-reported outcomes and the degree of concordance between physician and patient perceptions of symptom severity in advanced NSCLC in the USA. Patients with advanced (stage IIIB/IV) NSCLC (N = 450) were recruited in a nationwide (USA) lung cancer study. Patients and their oncologists completed patient and physician versions of the Lung Cancer Symptom Scale (LCSS). Patient-reported lung cancer-specific quality of life was assessed with the Functional Assessment of Cancer Therapy-Lung (FACT-L). Concordance was assessed using the kappa-statistic. Regression analysis was performed with FACT-L total score as the dependent variable and patient-reported LCSS symptom scores as predictors. A high proportion of patients experienced lung cancer symptoms: fatigue (100 %), loss of appetite (97 %), shortness of breath (95 %), cough (93 %), pain (92 %), and blood in sputum (63 %). Concordance between physician and patients was lowest for loss of appetite (kappa 0.1701) and greatest for hemoptysis (kappa 0.4586). Loss of appetite (β = -0.204; p < 0.001), cough (β = -0.145; p < 0.01), pain (β = -0.265; p < 0.001), and shortness of breath (β = -0.145; p < 0.01) were found to be significant predictors of the quality of life. Symptom burden in patients with advanced NSCLC is high and has a negative impact on the quality of life. Patient-reported outcomes data could help optimize disease outcomes and therapy management in NSCLC.Supportive Care in Cancer 09/2013; · 2.09 Impact Factor